scholarly journals CRISPR/Cas9-based genome engineering in HIV gene therapy

2021 ◽  
Vol 233 ◽  
pp. 02004
Author(s):  
Xuanting Tang
Keyword(s):  
Dna Cleavage ◽  
Genome Engineering ◽  
High Efficiency ◽  
Genetic Diseases ◽  
Adaptive Immune System ◽  
Research Progress ◽  
Specific Gene ◽  
Adaptive Immune ◽  
Efficiency Cost ◽  
Genetic Science

In recent years, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated nuclease (Cas) technology has become the most heated genome editing technique. Comparing to earlier genetic engineering methods, the CRISPR/Cas system is more advantageous due to its simple convenient design, high efficiency, cost-effectiveness, and the ability to perform multi-sites editing simultaneously. As the most effective gene editing tool, it utilizes a simple short RNA-guided mechanism to direct Cas-mediated DNA cleavage at the target genome locus and exploits the endogenous DNA repair pathways to achieve site-specific gene modifications. Initially discovered as a part of the bacterial adaptive immune system, the CRISPR/Cas system has now been widely used to study a broad range of biological genomes. Besides its contribution to our understanding of the basic genetic science, the application of the CRISPR/Cas system also expands rapidly into the medical fields, showing great potentials in the research of genetic diseases, viral infectious diseases, and cancers. In this review, the latest research progress of CRISPR/Cas technology is summarized based on its development, mechanism, and application in HIV/AIDS intervention. This review also examines the existing weaknesses and the future prospects of this promising technology.

scholarly journals Various Aspects of a Gene Editing System—CRISPR–Cas9

2020 ◽  
Vol 21 (24) ◽  
pp. 9604
Author(s):  
Edyta Janik ◽  
Marcin Niemcewicz ◽  
Michal Ceremuga ◽  
Lukasz Krzowski ◽  
Joanna Saluk-Bijak ◽  
...  
Keyword(s):  
Genome Editing ◽  
Gene Editing ◽  
Genome Engineering ◽  
High Efficiency ◽  
Adaptive Immune System ◽  
Zinc Finger Nucleases ◽  
Transcription Activator ◽  
Guide Rna ◽  
Adaptive Immune ◽  
Cas9 Protein

The discovery of clustered, regularly interspaced short palindromic repeats (CRISPR) and their cooperation with CRISPR-associated (Cas) genes is one of the greatest advances of the century and has marked their application as a powerful genome engineering tool. The CRISPR–Cas system was discovered as a part of the adaptive immune system in bacteria and archaea to defend from plasmids and phages. CRISPR has been found to be an advanced alternative to zinc-finger nucleases (ZFN) and transcription activator-like effector nucleases (TALEN) for gene editing and regulation, as the CRISPR–Cas9 protein remains the same for various gene targets and just a short guide RNA sequence needs to be altered to redirect the site-specific cleavage. Due to its high efficiency and precision, the Cas9 protein derived from the type II CRISPR system has been found to have applications in many fields of science. Although CRISPR–Cas9 allows easy genome editing and has a number of benefits, we should not ignore the important ethical and biosafety issues. Moreover, any tool that has great potential and offers significant capabilities carries a level of risk of being used for non-legal purposes. In this review, we present a brief history and mechanism of the CRISPR–Cas9 system. We also describe on the applications of this technology in gene regulation and genome editing; the treatment of cancer and other diseases; and limitations and concerns of the use of CRISPR–Cas9.

scholarly journals Comprehensive Mining and Characterization of CRISPR-Cas Systems in Bifidobacterium

2020 ◽  
Vol 8 (5) ◽  
pp. 720 ◽  
Author(s):  
Meichen Pan ◽  
Matthew A. Nethery ◽  
Claudio Hidalgo-Cantabrana ◽  
Rodolphe Barrangou
Keyword(s):  
Genome Engineering ◽  
Rare Variants ◽  
Adaptive Immune System ◽  
Effector Proteins ◽  
Type I ◽  
Human Gut ◽  
Adaptive Immune ◽  
Naturally Occurring ◽  
History Of

The clustered regularly interspaced short palindromic repeats (CRISPR)-Cas (CRISPR-associated cas) systems constitute the adaptive immune system in prokaryotes, which provides resistance against bacteriophages and invasive genetic elements. The landscape of applications in bacteria and eukaryotes relies on a few Cas effector proteins that have been characterized in detail. However, there is a lack of comprehensive studies on naturally occurring CRISPR-Cas systems in beneficial bacteria, such as human gut commensal Bifidobacterium species. In this study, we mined 954 publicly available Bifidobacterium genomes and identified CRIPSR-Cas systems in 57% of these strains. A total of five CRISPR-Cas subtypes were identified as follows: Type I-E, I-C, I-G, II-A, and II-C. Among the subtypes, Type I-C was the most abundant (23%). We further characterized the CRISPR RNA (crRNA), tracrRNA, and PAM sequences to provide a molecular basis for the development of new genome editing tools for a variety of applications. Moreover, we investigated the evolutionary history of certain Bifidobacterium strains through visualization of acquired spacer sequences and demonstrated how these hypervariable CRISPR regions can be used as genotyping markers. This extensive characterization will enable the repurposing of endogenous CRISPR-Cas systems in Bifidobacteria for genome engineering, transcriptional regulation, genotyping, and screening of rare variants.

scholarly journals CRISPR Cas9 Tip of an Iceberg: It’s Potential in Genome Editing and Many More

2021 ◽  
Author(s):  
Jalal Ahmad ◽  
Nayyer Siddique
Keyword(s):  
Genome Editing ◽  
Genetic Diseases ◽  
Adaptive Immune System ◽  
Biological Research ◽  
Adaptive Immune ◽  
Scientific World ◽  
A Genome ◽  
Foreign Dna ◽  
Immense Potential ◽  
Potential Use

Clustered regularly interspaced short palindromic repeats or CRISPR, one of the major technological tools from nature's toolbox, has revolutionized the scientific world with its potential use in humans and plants. CRISPR Cas9 was first known as an adaptive immune system of bacteria. It is a system that cleaves foreign DNA. It has been exploited to be used as a genome editing tool for correcting genetic diseases in humans, for plants to create stress-resistant plants, and for a variety of different purposes. This review provides a basic overview of its applications in different areas of biological research. It has immense potential for a variety of researches, but it's still a mystery for science. It feels like scientists just know a tip of an iceberg.

scholarly journals CRISPR-Cas9 in agriculture: Approaches, applications, future perspectives, and associated challenges

2020 ◽  
Vol 3 (1) ◽  
pp. 6-16
Author(s):  
Prabin Adhikari ◽  
Mousami Poudel
Keyword(s):  
Dna Cleavage ◽  
Gene Editing ◽  
Adaptive Immune System ◽  
Genetic Manipulations ◽  
Adaptive Immune ◽  
Cargo Delivery ◽  
Genome Wide ◽  
Rna Fragments ◽  
Sgrna Design ◽  
Living Organisms

AbstractThe discovery of an adaptive immune system especially in archae and bacteria, CRISPR/Cas has revolutionized the field of agriculture and served as a potential gene editing tool, producing great excitement to the molecular scientists for the improved genetic manipulations. CRISPR/Cas9 is a RNA guided endonuclease which is popular among its predecessors ZFN and TALEN’s. The utilities of CRISPR from its predecessors is the use of short RNA fragments to locate target and breaking the double strands which avoids the need of protein engineering, thus allowing time efficiency measure for gene editing. It is a simple, flexible and highly efficient programmable DNA cleavage system that can be modified for widespread applications like knocking out the genes, controlling transcription, modifying epigenomes, controlling genome-wide screens, modifying genes for disease and stress tolerance and imaging chromosomes. However, gene cargo delivery system, off target cutting and issues on the safety of living organisms imposes major challenge to this system. Several attempts have been done to rectify these challenges; using sgRNA design software, cas9 nickases and other mutants. Thus, further addressing these challenges may open the avenue for CRISPR/cas9 for addressing the agriculture related problems.

scholarly journals CRISPR/Cas9 and cancer

2021 ◽  
Vol 53 (2) ◽  
Author(s):  
Nora Mimoune ◽  
Mohamed Wail Bahouh ◽  
Said Boukhechem ◽  
Djamel Khelef ◽  
Rachid Kaidi
Keyword(s):  
Cancer Research ◽  
Clinical Trials ◽  
Immune System ◽  
Genome Engineering ◽  
Adaptive Immune System ◽  
Powerful Method ◽  
Efficient Technology ◽  
Adaptive Immune ◽  
Mouse Models Of Cancer ◽  
The Impact

CRISPR/Cas9 has become a powerful method for making changes to the genome of many organisms. First discovered in bacteria as part of an adaptive immune system, CRISPR/Cas9 and modified versions have found widespread use in genome engineering and in the activation or repression of gen expression. As such, CRISPR/Cas9 promises to accelerate cancer research by providing an efficient technology to dissect mechanisms of tumorigenesis, identify targets for drug development, and possibly arm cells for cell-based therapies. Here, we review the current applications of the CRISPR/Cas9 technology for cancer research and therapy. We highlight the impact of CRISPR/Cas9 in generating organoid and mouse models of cancer. Finally, we provide an overview of the first clinical trials applying CRISPR/Cas9 as a therapeutic approach against cancer.

scholarly journals Editor's cut: DNA cleavage by CRISPR RNA-guided nucleases Cas9 and Cas12a

2019 ◽  
Vol 48 (1) ◽  
pp. 207-219 ◽  
Author(s):  
Thomas Swartjes ◽  
Raymond H.J. Staals ◽  
John van der Oost
Keyword(s):  
Immune System ◽  
Genome Editing ◽  
Dna Cleavage ◽  
Adaptive Immune System ◽  
New Developments ◽  
Biochemical Characteristics ◽  
Base Editing ◽  
Dna Cleaving ◽  
Adaptive Immune ◽  
General Method

Discovered as an adaptive immune system of prokaryotes, CRISPR–Cas provides many promising applications. DNA-cleaving Cas enzymes like Cas9 and Cas12a, are of great interest for genome editing. The specificity of these DNA nucleases is determined by RNA guides, providing great targeting adaptability. Besides this general method of programmable DNA cleavage, these nucleases have different biochemical characteristics, that can be exploited for different applications. Although Cas nucleases are highly promising, some room for improvement remains. New developments and discoveries like base editing, prime editing, and CRISPR-associated transposons might address some of these challenges.

In utero gene transfer to the mouse nervous system

2010 ◽  
Vol 38 (6) ◽  
pp. 1489-1493 ◽  
Author(s):  
Ahad A. Rahim ◽  
Andrew M.S. Wong ◽  
Suzanne M.K. Buckley ◽  
Jerry K.Y. Chan ◽  
Anna L. David ◽  
...  
Keyword(s):  
Nervous System ◽  
Gene Transfer ◽  
Progenitor Cell ◽  
Muscular Atrophy ◽  
Genetic Diseases ◽  
Adaptive Immune System ◽  
Early Gene ◽  
Fetal Mouse ◽  
Adaptive Immune ◽  
Technological Perspective

The cellular and molecular environment present in the fetus and early newborn provides an excellent opportunity for effective gene transfer. Innate and pre-existing anti-vector immunity may be attenuated or absent and the adaptive immune system predisposed to tolerance towards xenoproteins. Stem cell and progenitor cell populations are abundant, active and accessible. In addition, for treatment of early lethal genetic diseases of the nervous system, the overarching advantage may be that early gene supplementation prevents the onset of irreversible pathological changes. Gene transfer to the fetal mouse nervous system was achieved, albeit inefficiently, as far back as the mid-1980s. Recently, improvements in vector design and production have culminated in near-complete correction of a mouse model of spinal muscular atrophy. In the present article, we review perinatal gene transfer from both a therapeutic and technological perspective.

scholarly journals Oligosaccharides: Defense Inducers, Their Recognition in Plants, Commercial Uses and Perspectives

Molecules ◽  
2020 ◽  
Vol 25 (24) ◽  
pp. 5972
Author(s):  
Nathalie Guarnizo ◽  
Diego Oliveros ◽  
Walter Murillo-Arango ◽  
María Bianney Bermúdez-Cardona
Keyword(s):  
Defense Mechanisms ◽  
High Efficiency ◽  
Adaptive Immune System ◽  
Innate Immune ◽  
Pathogenic Microorganisms ◽  
Commercial Products ◽  
Resistance Induction ◽  
Adaptive Immune ◽  
Cellular Events ◽  
Increase Productivity

Plants have innate immune systems or defense mechanisms that respond to the attack of pathogenic microorganisms. Unlike mammals, they lack mobile defense cells, so defense processes depend on autonomous cellular events with a broad repertoire of recognition to detect pathogens, which compensates for the lack of an adaptive immune system. These defense mechanisms remain inactive or latent until they are activated after exposure or contact with inducing agents, or after the application of the inductor; they remain inactive only until they are affected by a pathogen or challenged by an elicitor from the same. Resistance induction represents a focus of interest, as it promotes the activation of plant defense mechanisms, reducing the use of chemical synthesis pesticides, an alternative that has even led to the generation of new commercial products with high efficiency, stability and lower environmental impact, which increase productivity by reducing not only losses but also increasing plant growth. Considering the above, the objective of this review is to address the issue of resistance induction with a focus on the potential of the use of oligosaccharides in agriculture, how they are recognized by plants, how they can be used for commercial products and perspectives.

scholarly journals On-Target CRISPR/Cas9 Activity Can Cause Undesigned Large Deletion in Mouse Zygotes

2020 ◽  
Vol 21 (10) ◽  
pp. 3604 ◽  
Author(s):  
Alexey Korablev ◽  
Varvara Lukyanchikova ◽  
Irina Serova ◽  
Nariman Battulin
Keyword(s):  
Genome Engineering ◽  
Adaptive Immune System ◽  
Strand Break ◽  
Large Deletion ◽  
Dna Breaks ◽  
Adaptive Immune ◽  
Double Strand Dna Breaks ◽  
Mouse Zygotes ◽  
Genomic Editing ◽  
Cytoplasmic Injection

Genome engineering has been tremendously affected by the appearance of the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (CRISPR/Cas9)-based approach. Initially discovered as an adaptive immune system for prokaryotes, the method has rapidly evolved over the last decade, overtaking multiple technical challenges and scientific tasks and becoming one of the most effective, reliable, and easy-to-use technologies for precise genomic manipulations. Despite its undoubtable advantages, CRISPR/Cas9 technology cannot ensure absolute accuracy and predictability of genomic editing results. One of the major concerns, especially for clinical applications, is mutations resulting from error-prone repairs of CRISPR/Cas9-induced double-strand DNA breaks. In some cases, such error-prone repairs can cause unpredicted and unplanned large genomic modifications within the CRISPR/Cas9 on-target site. Here we describe the largest, to the best of our knowledge, undesigned on-target deletion with a size of ~293 kb that occurred after the cytoplasmic injection of CRISPR/Cas9 system components into mouse zygotes and speculate about its origin. We suppose that deletion occurred as a result of the truncation of one of the ends of a double-strand break during the repair.

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